Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD

NCT04704921 | Active Not Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: RGX-314-2104M23-416
• Study Type: interventional
• Target: 671
• Locations: 1 country
• Sites: 89

Brief Summary

ABBV-RGX-314 (also known as RGX-314) is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (wet AMD or nAMD). Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-vascular endothelial growth factor (anti-VEGF) therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to maintain or prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every 4 to 16 weeks in frequency, to maintain efficacy. Due to the burden of these treatments, patients often experience a decline in vision with reduced frequency of treatment over time.

Conditions

AMD; nAMD; wAMD; Wet Age-related Macular Degeneration; Wet AMD; CNV; Neovascular AMD; Choroidal Neovascularization; Neovascular Age-related Macular Degeneration

Keywords

Age-Related Macular Degeneration; Neovascular Age-Related Macular Degeneration; Macular Degeneration; Wet Macular Degeneration; Choroidal Neovascularization; Retinal Degeneration; Retinal Diseases; Eye Diseases; Ranibizumab; Angiogenesis Inhibitors; Angiogenesis Modulating Agents; Growth Substances; Physiological Effects of Drugs; Growth Inhibitors; Antineoplastic Agents; Gene therapy; Anti-vascular endothelial grown factory therapy; Anti-VEGF therapy

Outcome Measures

Primary Outcomes (2)

• Mean change from baseline in Best Corrected Visual Acuity (BCVA)

  BCVA measured by Early Treatment Diabetic Retinopathy Study (ETDRS)

  At Week 54

• Bilateral Treatment Substudy: Incidence of ocular AEs and any SAEs

  Incidence of ocular AEs and any SAEs

  Week 50

Secondary Outcomes (38)

• Incidences of ocular and overall AEs over 54 weeks

  Through Week 54

• Incidences of ocular and overall AEs over 98 weeks

  Through Week 98

• Mean change from baseline in BCVA to Week 98 (ABBV-RGX-314 randomized participants) based on the ETDRS score

  Week 98

• Proportion of participants with worsened BCVA

  Week 54; Week 98

• Proportion of participants with improved BCVA

  Week 54; Week 98

Study Arms (3)

ABBV-RGX-314 Dose 1

EXPERIMENTAL

ABBV-RGX-314 Dose 1 administered via subretinal delivery one time.

Genetic: ABBV-RGX-314

ABBV-RGX-314 Dose 2

EXPERIMENTAL

ABBV-RGX-314 Dose 2 administered via subretinal delivery one time.

Genetic: ABBV-RGX-314

Control Arm

ACTIVE COMPARATOR

Ranibizumab administered via intravitreal injection approximately every 28 days

Biological: Ranibizumab (LUCENTIS®)

Interventions

ABBV-RGX-314 GENETIC

AAV8 vector containing a transgene for anti-VEGF Fab (Dose 1)

Also known as: surabgene lomparvovec, RGX-314

ABBV-RGX-314 Dose 1

Ranibizumab (LUCENTIS®) BIOLOGICAL

0.5 mg (0.05 mL of 10 mg/mL solution) administered by intravitreal injection approximately every 28 days

Also known as: Ranibizumab (anti-VEGF agent)

Control Arm

Eligibility Criteria

• Age: 50 Years - 89 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 50 years and ≤ 89 years
• An ETDRS BCVA letter score between ≤ 78 and ≥ 40 in the study eye
• Diagnosis of subfoveal CNV secondary to AMD in the study eye previously treated with anti-VEGF
• Must be pseudophakic (at least 12 weeks postcataract surgery) in the study eye.
• Willing and able to provide written, signed informed consent for this study
• Participants must have demonstrated a meaningful response to anti-VEGF therapy at study entry
• An ETDRS BCVA letter score between ≤ 83 and ≥ 40 in both eyes
• Diagnosis of subfoveal choroidal neovascularization (CNV) secondary to AMD in both eyes
• Must be pseudophakic (at least 12 weeks postcataract surgery) in both eyes
• Willing and able to provide written, signed informed consent for this study
• Newcomers must have active disease in the study eye; crossover participants must have active disease in the eye not treated in the main study

You may not qualify if:

• CNV or macular edema in the study eye secondary to any causes other than AMD
• Subfoveal fibrosis or atrophy in the study eye, as determined by CRC
• Any condition in the investigator's opinion that could limit VA improvement in the study eye
• Active or history of retinal detachment, or current retinal tear that cannot be treated, in the study eye
• Advanced glaucoma or history of secondary glaucoma in the study eye
• History of intraocular surgery in the study eye within 12 weeks prior to randomization
• History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational product, other than anti-VEGF therapy, in the 6 months prior to Screening Visit 1
• Prior treatment with gene therapy
• Recent myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months
• CNV or macular edema in either eye secondary to any causes other than AMD
• Subfoveal fibrosis or atrophy in either eye
• Any condition in the investigator's opinion that could limit VA improvement in either eye
• Active or history of retinal detachment, or current retinal tear that cannot be treated in either eye
• Advanced glaucoma or history of secondary glaucoma in either eye
• Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months

Sponsors & Collaborators

• REGENXBIO Inc.: collaborator
• AbbVie: lead

Study Sites (89)

Retinal Research Institute /ID# 256019

Phoenix, Arizona, 85053, United States

Location

Barnet Dulaney Perkins Eye Center - Sun City /ID# 256055

Sun City, Arizona, 85351, United States

Location

University of Arkansas for Medical Sciences /ID# 271290

Little Rock, Arkansas, 72205, United States

Location

Retina Vitreous Assoc Med Grp /ID# 256299

Beverly Hills, California, 90211, United States

Location

Retinal Diagnostic Center /ID# 256137

Campbell, California, 95008, United States

Location

The Retina Partners - Encino /ID# 256054

Encino, California, 91436, United States

Location

Retina Consultants of Orange County /ID# 256152

Fullerton, California, 92835, United States

Location

Salehi Retina Institute /ID# 263485

Huntington Beach, California, 92647, United States

Location

UC Irvine/Gavin Herbert Eye Institute /ID# 256145

Irvine, California, 92697, United States

Location

Northern California Retina Vitreous Associates Medical Group, Inc /ID# 256298

Mountain View, California, 94040-4101, United States

Location

UCLA Doheny Eye Center /ID# 256120

Pasadena, California, 91105, United States

Location

California Eye Specialists Medical Group Inc. /ID# 256079

Pasadena, California, 91107, United States

Location

Retina Consultants of San Diego /ID# 256021

Poway, California, 92064-2530, United States

Location

Retinal Consultants Medical Group /ID# 256047

Sacramento, California, 95825, United States

Location

West Coast Retina /ID# 256448

San Francisco, California, 94107, United States

Location

University of California, San Francisco /ID# 256130

San Francisco, California, 94143, United States

Location

Orange County Retina Medical Group /ID# 256073

Santa Ana, California, 92705, United States

Location

California Retina Consultants - Santa Barbara /ID# 256017

Santa Barbara, California, 93103, United States

Location

Retina Consultants of Southern Colorado /ID# 256069

Colorado Springs, Colorado, 80909, United States

Location

Southwest Retina Research Center /ID# 256136

Durango, Colorado, 81303, United States

Location

Colorado Retina Associates /ID# 256121

Lakewood, Colorado, 80228, United States

Location

Retina Group of New England - Waterford /ID# 256071

Waterford, Connecticut, 06385-1215, United States

Location

Advanced Research, LLC /ID# 275451

Deerfield Beach, Florida, 33064-1342, United States

Location

Vitreoretinal Associates, P.A. /ID# 256150

Gainesville, Florida, 32607, United States

Location

Florida Retina Consultants /ID# 265661

Lakeland, Florida, 33805, United States

Location

Bascom Palmer Eye Institute - University of Miami /ID# 256072

Miami, Florida, 33136, United States

Location

Retina Specialty Institute /ID# 256153

Pensacola, Florida, 32503, United States

Location

Retina Vitreous Associates of Florida - St. Petersburg /ID# 256050

St. Petersburg, Florida, 33711-1141, United States

Location

Southern Vitreoretinal Associates /ID# 256158

Tallahassee, Florida, 32308, United States

Location

Southeast Retina Center /ID# 256022

Augusta, Georgia, 30909, United States

Location

Georgia Retina - Marietta /ID# 256142

Marietta, Georgia, 30060, United States

Location

Marietta Eye Clinic /ID# 268163

Marietta, Georgia, 30060, United States

Location

Thomas Eye Group PC /ID# 268159

Sandy Springs, Georgia, 30328-4411, United States

Location

Retina Consultants of Hawaii /ID# 256049

‘Aiea, Hawaii, 96701, United States

Location

University of Illinois at Chicago /ID# 256300

Chicago, Illinois, 60607, United States

Location

University Retina and Macula Associates /ID# 256078

Lemont, Illinois, 60439, United States

Location

University Retina and Macula Associates /ID# 256077

Oak Forest, Illinois, 60452, United States

Location

Springfield Clinic /ID# 266225

Springfield, Illinois, 62702-3749, United States

Location

Retina Partners Midwest, PC /ID# 256045

Indianapolis, Indiana, 46290, United States

Location

John-Kenyon American Eye Institute -New Albany /ID# 256065

New Albany, Indiana, 47150-3620, United States

Location

Retina Associates - Lenexa /ID# 256080

Lenexa, Kansas, 66215, United States

Location

Cincinnati Eye Institute- Edgewood /ID# 256132

Edgewood, Kentucky, 41017-3415, United States

Location

Retina Associates of New Orleans /ID# 272440

Metairie, Louisiana, 70006-2940, United States

Location

Ochsner Medical Center - Jefferson Highway /ID# 270524

New Orleans, Louisiana, 70121, United States

Location

The Retina Care Center /ID# 256144

Baltimore, Maryland, 21209, United States

Location

Johns Hopkins Hospital /ID# 256015

Baltimore, Maryland, 21287, United States

Location

The Retina Group Of Washington - Chevy Chase /ID# 276039

Chevy Chase, Maryland, 20815, United States

Location

Cumberland Valley Retina Consultants - Hagerstown /ID# 256151

Hagerstown, Maryland, 21740, United States

Location

Ophthalmic Consultants of Boston /ID# 256014

Boston, Massachusetts, 02114, United States

Location

Retina Associates of Michigan /ID# 266198

Grand Blanc, Michigan, 48439, United States

Location

Associated Retinal Consultants /ID# 256156

Royal Oak, Michigan, 48073, United States

Location

VitreoRetinal Surgery PLLC DBA Retina Consultants of Minnesota /ID# 256160

Edina, Minnesota, 55435, United States

Location

Mayo Clinic - Minnesota /ID# 256051

Rochester, Minnesota, 55905, United States

Location

The Retina Institute /ID# 266587

St Louis, Missouri, 46214, United States

Location

Sierra Eye Associates /ID# 256020

Reno, Nevada, 89502, United States

Location

Eye Associates of New Mexico /ID# 256075

Albuquerque, New Mexico, 87109, United States

Location

Long Island Vitreoretinal Consultants /ID# 256074

Great Neck, New York, 11021, United States

Location

Retina-Vitreous Surgeons of Central New York - Liverpool /ID# 266274

Liverpool, New York, 13088, United States

Location

Duke Eye Center /ID# 256076

Durham, North Carolina, 27705, United States

Location

Atrium Health Wake Forest Baptist Medical Center /ID# 270767

Winston-Salem, North Carolina, 27157, United States

Location

Retina Associates of Cleveland-Middleburg Heights /ID# 256052

Cleveland, Ohio, 44130, United States

Location

Cleveland Clinic Main Campus /ID# 256064

Cleveland, Ohio, 44195, United States

Location

Retina Vitreous Center, Research /ID# 256067

Edmond, Oklahoma, 73013, United States

Location

Verum Research, LLC /ID# 266585

Eugene, Oregon, 97401, United States

Location

Retina Northwest, PC /ID# 256140

Portland, Oregon, 97221, United States

Location

Erie Retina Research /ID# 256154

Erie, Pennsylvania, 16507, United States

Location

Retina Vitreous Consultants - Monroeville /ID# 271654

Monroeville, Pennsylvania, 15146, United States

Location

Mid Atlantic Retina /ID# 256013

Philadelphia, Pennsylvania, 19107, United States

Location

Charleston Neuroscience Institute /ID# 256235

Ladson, South Carolina, 29456, United States

Location

Black Hills Regional Eye Institute /ID# 256161

Rapid City, South Dakota, 57701, United States

Location

Charles Retina Institute /ID# 256016

Germantown, Tennessee, 38138, United States

Location

Retina Research Institute of Texas /ID# 256141

Abilene, Texas, 79606-1224, United States

Location

Austin Research Center for Retina /ID# 256148

Austin, Texas, 78705, United States

Location

Austin Retina Associates - Austin /ID# 256053

Austin, Texas, 78705, United States

Location

Austin Clinical Research, LLC /ID# 256043

Austin, Texas, 78750-2298, United States

Location

Retina Consultants of Texas - Beaumont /ID# 266279

Beaumont, Texas, 77707, United States

Location

Retina and Vitreous of Texas /ID# 263961

Bellaire, Texas, 77401, United States

Location

Texas Retina Associates - Dallas /ID# 256133

Dallas, Texas, 75231, United States

Location

Baylor Scott & White Surgicare /ID# 256122

Fort Worth, Texas, 76104, United States

Location

Retina Consultants Of Texas /ID# 268611

San Antonio, Texas, 78240, United States

Location

Stone Oak Surgery Center /ID# 266199

San Antonio, Texas, 78240, United States

Location

Retina Center Of Texas (Rct) - Southlake /ID# 256056

Southlake, Texas, 76092, United States

Location

Retina Consultants - The Woodlands /ID# 256018

The Woodlands, Texas, 77384, United States

Location

Retina Associates of Utah /ID# 256044

Salt Lake City, Utah, 84107, United States

Location

John A. Moran Eye Center /ID# 256068

Salt Lake City, Utah, 84132, United States

Location

Wagner Macula & Retina Center - Norfolk /ID# 256134

Norfolk, Virginia, 23502, United States

Location

Pacific Northwest Retina /ID# 256155

Bellevue, Washington, 98004, United States

Location

Retina Center NW, PLLC /ID# 256157

Silverdale, Washington, 98383, United States

Location

University of Wisconsin-Madison, Department of Ophthalmology and Visual Sciences /ID# 256159

Madison, Wisconsin, 53705-3644, United States

Location

Related Links

• Related Info

MeSH Terms

Conditions

Choroidal Neovascularization; Macular Degeneration; Wet Macular Degeneration; Retinal Degeneration; Retinal Diseases; Eye Diseases

Interventions

Ranibizumab

Condition Hierarchy (Ancestors)

Choroid Diseases; Uveal Diseases; Neovascularization, Pathologic; Metaplasia; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Eye Diseases, Hereditary

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• ABBVIE INC.

  AbbVie

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: The administration of ABBV-RGX-314 requires an outpatient surgical procedure performed in an operating room, while the active control, ranibizumab, is administered via intravitreal injection in an office setting. This study will be partially masked which will include masking of key study assessors and study drug dose.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 2 ABBV-RGX-314 treatment arms, 1 control arm (ranibizumab)

Study Record Dates

• First Submitted: January 8, 2021
• First Posted: January 12, 2021
• Study Start: December 29, 2020
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): November 1, 2027
• Last Updated: April 28, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (89)